“That’s OK, I’ll Be Patient #1”
How spinal cord injury met its match
Clinical trials are the standard (classic, normal) way in which patients obtain access to investigational drugs and therapies. They are typically designed for a group of patients, more often called subjects. A trial sponsor is usually the entity that sets up the trial, pays for it and is responsible for reporting the data. Any sponsor can testify about how much time and energy it takes to design and execute a trial.
For the Hope Biosciences Stem Cell Research Foundation (HBSCRF), sponsoring trials is what we do. But what happens when a patient comes to us and there are no trials for which they can qualify? It is in those situations sponsors have 2 alternative options. The first one is FDA’s expanded access program. It is designed for patients who have exhausted all existing medical options and don’t have a clinical trial that they can participate in. The second option is the “Right to Try” program. This new program was signed into law in 2019 and allows terminally ill patients to have access to investigational drugs and therapies. The justification for the creation of this bill was largely due to the enormous burden and time expanded access applications had for the sponsor. Many sponsors simply refused to make their therapies available outside of a standard clinical trial because expanded access protocols were too time consuming and expensive to execute. In an effort to encourage sponsors to make their therapies available, the Right-to-Try program would eliminate the burden of writing protocols, investigational new drug applications, getting IRB approvals and receiving FDA authorizations.
HBSCRF made the difficult decision not to make the Right-To-Try option available. It was something that we heavily contemplated, but in the end, decided it simply did not serve the greater good. When clinical trials or expanded access protocols are executed, we are literally serving the patient being treated. But the end goal is to observe the efficacy of a treatment, report it and get it approved so EVERYBODY can have access to it. It’s so difficult to compartmentalize, but if you can look beyond the face of the patient you are treating, you will be able to see the millions of faces behind them that are depending on you to get the data right.
Creating an expanded access protocol for a single patient takes just as much time as it does for a standard clinical trial. It still requires us to create a scientific justification, support it with preclinical data and create a protocol that can pass FDA and an IRB’s review. These steps, however long they take, are necessary not only to ensure patient safety but to allow us to collect the right data. EVERY DATA POINT COUNTS. Even if that data point is insignificant for regulatory approval, it can be instrumental in understanding how to design the next big trial — the one that LEADS to approval.
Michael is 19 years old. On Christmas Eve of 2020, a skiing accident left him with a completely transected spinal cord at level T10–11. Michael’s parents reached out to us for help and our team jumped right in. While he was in the hospital, his doctor warned him that there was no evidence that stem cells would help him and Michael replied, “That’s ok, I’ll be patient #1”.
Today, March 11, 2021, 77 days post injury, we received FDA authorization to start his very own trial. We do have other spinal cord injury protocols but this is the first time we will attempt to treat a complete injury. To our knowledge, this is the first time, globally, that this kind of injury will be treated with the magnitude of doses that we have been authorized. We will begin treatments in the coming weeks and we will be sharing results in the near future. As researchers, it is in our nature to be somewhere between skeptical and cautiously optimistic. Regardless of the sentiment, the fact is, every known cure started with patient #1.
